MedTrace Logo

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 66

Last updated 2023-08-29

Study results available
· View outcomes & findings →

Summary

Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here.

This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords).

In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control).

In Part C, all participants will receive study drug.

Conditions

  • Niemann-Pick Disease, Type C

Interventions

DRUG

Adrabetadex

Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.

Sponsors & Collaborators

  • Mandos LLC

    lead INDUSTRY

Principal Investigators

  • Clinical Study Lead · Mandos LLC

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-12-23
Primary Completion
2022-04-11
Completion
2022-04-11
FDA Drug
Yes

Countries

  • United States
  • Australia
  • France
  • Germany
  • New Zealand
  • Singapore
  • Switzerland
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04958642 on ClinicalTrials.gov